With more than two million page views and more than 4,500 items, this blog provides news and commentary on public policy, business and economic issues related to the $3 billion California stem cell agency, officially known as the California Institute for Regenerative Medicine(CIRM). David Jensen, a retired California newsman, has published this blog since January 2005. His email address is djensen@californiastemcellreport.com.

Monday, January 12, 2015

California’s $3 billion stem cell agency this month is
kicking off the first round of radical changes in how it finances research,
including funding of the clinical trials that it hopes will push nascent
therapies into widespread use.

The money is scheduled to start flowing to scientists in the
Golden State as soon as June in a new, fast-track program that is called CIRM
2.0. But only if the researchers file their applications within
the next 19 days. Slow-moving
researchers, however, will have a chance to file again at the end of next month
and each following month.

The effort has all the earmarks of a venture capital program
with heavy involvement by the financing party (CIRM), involving both the budget
and the science. The agency, known as CIRM after its formal name of the California Institute for Regenerative
Medicine, last Friday repeated its pledge to be an “active investor,” which may
or may not be welcomed by some researchers.

Nonetheless, Los Angeles Times columnist Michael Hiltzik
recently wrote that scientists would find a “lot to like” in the new direction
at the agency. Here is how CIRM put it in a news release Dec. 31 that contained
links to the detailed program announcements,

“CIRM 2.0 is a radical overhaul of the way the agency does
business, implementing efficient new systems and programs that place added
emphasis on speed, partnerships, and patients.

“CIRM 2.0 makes it easier for both companies and academic
researchers with promising projects to partner with CIRM to get the support
they need when they need it, reducing the time from application to funding from
around two years to just 120 days.”

The release laid out some of the aspects of the new grant review
and funding process, which the agency expects to extend to all its remaining $1 billion in award
programs,

“Speed: In addition in to reducing the time to funding
to 120 days, new clinical stage projects may be submitted to CIRM year round
instead of only once or twice a year as in the past. Applications simply have
to be filed by 5pm PT on the last business day of the month to be eligible for
consideration in that round of review. If you miss the deadline one month, you
only have to wait 30 days for the next one.

“Partnerships: Under CIRM 2.0, the agency will not act
as a passive funding source, but instead will be an active investor, devoting
significant internal resources and leveraging its vast external team of
world-class subject matter experts to advance the projects it selects. This
will result in a true partnership that both accelerates programs and gives them
the greatest opportunity for success.

“Patients: Each project will be partnered with a
project-specific Clinical Advisory Panel (CAP) to help advise and guide
it forward. Importantly, every panel will include at least one patient
advisor with first-hand experience of the specific condition, who will provide
input, recommendations and the appropriate sense of urgency that can only come
from the unique perspective of someone living with the disease.”

The agency has allotted $50 million to be handed out between
now and July for programs involving clinical trials or preparation for clinical
trials. Applicants can ask for as much
cash as they desire. But their proposed budgets will be scrutinized by an
outside panel and subject to possibly substantial revision. If CIRM does not
fancy the researcher’s figures, the application will not be presented to
reviewers.

The budget review is a significant change from past
procedures. It is just one of many
changes, small and large, that scientists need to understand about the new
process. Others involve restrictions on appeals, solvency requirements for businesses
and, in some cases, hefty co-funding requirements that must be documented in
advance. In-kind support, for example, will no longer be acceptable as matching cash or for co-funding.

If scientists want to be competitive, they would be wise to
carefully review the program announcements posted at the first of the month. In
some cases in years past, applicants have lost out simply because they failed
to understand in a timely fashion all the nuances of the process.

Applicants must be ready to work within 45 days of CIRM
board approval of their applications.

The agency – not grant reviewers -- will determine whether
applications meet eligibility requirements. If they do not, they will not be
reviewed. An appeal by an applicant may be made behind closed doors to the grant
review group.

No public appeals are formally allowed. However, state law
permits all members of the public, including researchers, to speak directly to
the board on any issue. That avenue for seeking funding, however, has not been
very successful in recent months.

The grant review group is likely to continue to make -- behind
closed doors -- virtually all the de facto decisions on application approvals.
Their actions will come in three "recommendations" to the board: fund the
application, do not fund but allow re-submission or do not fund and do not allow
resubmission. The CIRM board will then
ratify in public the group’s decision, as it has almost invariably in the past.
The board has the legal authority to do anything it wants with applications,
but it has rarely overturned positive decisions by the board. Infrequently, it
would approve some lower ranked applications. The new procedures would seem to make any public appeals to the board by
researchers even less likely to be successful.

On Jan. 20, the CIRM directors Science Subcommittee is scheduled
to vote on new grant administration rules involving CIRM 2.0. That is another
matter that researchers who wish to be successful in securing funds should be
following and making recommendations on. Sites where they can participate are in San
Francisco, Oakland, Irvine, Duarte, La Jolla and Sacramento. See the agenda for specific addresses.

Gil Sambrano continues to head the review process, albeit
with a new title: director of portfolio development and review. His phone
number and email can be found on the program announcements posted at the first
of the month (see here, here and here).

About Me

The California Stem Cell Report is the only nongovernmental website devoted solely to the $3 billion California stem cell agency. The report is published by David Jensen, who worked for 22 years for The Sacramento Bee in a variety of editing positions, including executive business editor and special projects editor. He was the primary editor on the 1992 Pulitzer Prize-winning series, "The Monkey Wars" by Deborah Blum, which dealt with opposition to research on primates. Jensen served as a press aide in the 1974 campaign and first administration of Gov. Jerry Brown. (Time served: two years and one week.) He writes from his sailboat on the west coast of Mexico with occasional visits to land. Jensen began writing about the stem cell agency in 2005, noting that it is an unprecedented effort that uniquely combines big science, big business, big academia, big politics, religion, ethics and morality as well as life and death. The California Stem Cell Report has been identified as one of the best stem cell sites on the Internet. Its readership includes the media (both mainstream and science), a wide range of academic/research institutions globally, the NIH and California policy makers.